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Why build a gene therapy around a blood stem cell instead of a virus alone?
A hematopoietic stem cell (HSC) — the cell that gives rise to your entire blood and immune system — can be corrected once, outside the body, and then put back in. It engrafts permanently, and its corrected descendants keep making the missing enzyme for years.
That's the foundation Live-cel is built on: harvest, correct, re-infuse. No repeat dosing of a virus. No permanent antibody response blocking a second treatment.
For the scientists and clinicians here: what's the biggest open question you'd want answered about HSC-based gene therapy durability? Genuinely curious where you'd focus.
Follow the whole series: https://bit.ly/Glafabra-TTW
@Brian Christie @Neva West @Chris Hopkins @Tetsu Yung
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